FDA OKs Apremilast (Otezla ) for Oral Ulcers of Behcet's Disease

Megan Brooks

July 22, 2019

The US Food and Drug Administration (FDA) has approved the use of apremilast (Otezla, Celgene) 30 mg twice daily for the treatment of adults with oral ulcers associated with Behçet's disease, the company has announced.

Behçet's disease, also known as Behçet's syndrome, affects roughly 5 in 100,000 people in the United States. Oral ulcers are the most common manifestation of the disease.

"Oral ulcers are a recurring and debilitating manifestation that affects nearly everyone living with Behçet's disease, and have an important negative impact on the quality of life for these patients," Yusuf Yazici, MD, clinical associate professor, Department of Medicine, New York University Langone Health, New York City, said in a company news release

Apremilast is an oral phosphodiesterase-4 (PDE-4) inhibitor that decreases proinflammatory mediators and increases anti-inflammatory mediators. It is the only FDA-approved treatment for oral ulcers associated with Behçet's disease.

Approval was based on efficacy and safety results from the randomized, placebo-controlled, double-blind phase 3 RELIEF study. Participants included 207 adults with Behçet's disease and active oral ulcers who were previously treated with at least one nonbiologic medication and were candidates for systemic therapy.

Treatment with apremilast 30 mg twice daily led to a 42.7-point reduction from baseline in the pain of oral ulcers as measured by the visual analog scale (VAS) at week 12 compared with an 18.7-point reduction with placebo. Significantly more patients in the apremilast group were free of oral ulcers at week 12 (52.9% vs 22.3%).  

In addition, patients taking apremilast had fewer daily ulcers during the 12-week study (1.5 on average vs 2.6 in the placebo group).

This approval "marks an important milestone for people with Behçet's disease who have been eagerly waiting for treatment options for their oral ulcers," Mirta Avila Santos, MD, executive director of the American Behçet's Disease Association, said in the release.

In the study, the most common adverse events associated with apremilast were diarrhea (41.3%), nausea (19.2%), headache (14.4%), and upper respiratory tract infection (11.5%). The safety profile was consistent with the known safety profile of apremilast.

Apremilast is already approved for patients with moderate to severe plaque psoriasis who are candidates for phototherapy or systemic therapy, and for adult patients with active psoriatic arthritis.

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